Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

Optimized genome-editing method opens the door to more effective treatment of genetic diseases

CRISPR-Cas9 is widely used to edit the genome by studying genes of interest and modifying disease-associated genes.

11 Apr 2023

3D structure of a probable precursor to the CRISPR-Cas12 enzyme revealed

Gene editing is one of the latest breakthroughs in biology. The widely known CRISPR-Cas gene editing system provided prokaryotes (organisms that lack cell nuclei) an immunity against foreign DNA. Since the discovery of the CRISPR gene editing technology, scientists are revealing the evolution of the CRISPR-Cas proteins from their precursors.

7 Apr 2023

Using brain organoids to test gene therapies for a rare disease in children

Brain tissue grown in a laboratory by University of Queensland researchers will be used to test a treatment for a rare disease in children and help unlock therapies for a range of neurological disorders.

5 Apr 2023

Complex assembly process involved in DNA virus replication revealed

In a twist on the question, "Which came first, the chicken or the egg?", scientists have long faced a similar question about how human adenovirus replicates: "Which comes first, assembly of the viral particle, or packaging of the viral genome?"

5 Apr 2023

Research provides new insights into the underlying disease mechanisms for polycystic kidney disease

Autosomal dominant polycystic kidney disease (ADPKD) is the most common potentially lethal genetic disease-;about a half million people in the United States alone suffer from the condition.

4 Apr 2023

Utilizing a molecular syringe for the delivery of functional proteins into human cells with high efficiency

Researchers investigated the feasibility of utilizing a bacterial contractile injection system for programmable protein delivery.

31 Mar 2023

Riboswitch may be the key to advance gene therapy, regenerative medicine

The Okinawa Institute of Science and Technology, in collaboration with Astellas Pharma Inc., has developed a new toolkit that uses small molecules to control the activity of a piece of synthetic RNA, and ultimately regulate gene expression.

30 Mar 2023

Lab-made antibodies may be able to cure people infected with yellow fever

New research from Oregon Health & Science University and collaborators indicates lab-made antibodies may be able to cure people infected with yellow fever, a virus for which there is no treatment.

29 Mar 2023

Mouse avatars could help design more effective and personalized therapies for multiple myeloma

An international group of researchers, led by Dr. José Ángel Martínez-Climent at the Cima University of Navarra, has created mouse avatars of patients with multiple myeloma to study and develop personalized treatments against this blood cancer, the second most frequent hematological cancer and incurable in most cases.

29 Mar 2023

FDA-approved drug keeps light-sensitive photoreceptors alive in lab models of LCA 10

A National Institutes of Health team has identified a compound already approved by the U.S. Food and Drug Administration that keeps light-sensitive photoreceptors alive in three models of Leber congenital amaurosis type 10 (LCA 10), an inherited retinal ciliopathy disease that often results in severe visual impairment or blindness in early childhood.

29 Mar 2023

Developers of gene therapies can utilize FDA guidance to prepare ODD or RPDD applications

Providing an overview of the submissions process and examples of U.S. Food and Drug Administration (FDA) applications for Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD), a new article can help developers of gene therapies for rare genetic diseases.

27 Mar 2023

Discovery suggests a new way to treat spinal muscular atrophy

Columbia researchers have discovered how a genetic defect leads to spinal muscular atrophy (SMA), a critical piece of information about the disease that neurologists have been seeking for decades.

23 Mar 2023

Disruption of the FcRn–IgG interaction may make AAV-based gene therapies effective

The use of a monoclonal antibody that reduced circulating IgG levels, led to a decrease in preexisting neutralizing antibodies (NAbs) to adeno-associated virus (AAV).

22 Mar 2023

Snapping at new biotech breakthroughs with Gator Pro

In this interview, we speak to Subodh Nimkar, Marketing Director at Gator Bio, about their latest technology, Gator Pro which allows for fast, reliable, and high-quality data.

From Gator Bio, Inc. 21 Mar 2023

Study highlights a persistent life expectancy gap among patients living with sickle cell disease

While research has long established disparities in health outcomes among individuals living with sickle cell disease (SCD), few studies have quantified these gaps.

16 Mar 2023

Study provides strong support for AAV.NT-3 gene therapy in the management of sarcopenia

A new research paper was published on the cover of Aging (listed by MEDLINE/PubMed as "Aging (Albany NY)" and "Aging-US" by Web of Science) Volume 15, Issue 5, entitled, "AAV1.NT-3 gene therapy prevents age-related sarcopenia."

16 Mar 2023

University of Utah and Tikkunlev partner to bring heart failure gene therapy innovation to the clinic

The University of Utah, one of the nation's leading research universities, and TikkunLev Therapeutics, a gene therapy company, have announced a new partnership to accelerate an innovative heart-failure gene therapy.

8 Mar 2023

Common scientific method to analyze DNA in early human embryos fails to accurately reflect gene edits

A commonly used scientific method to analyze a tiny amount of DNA in early human embryos fails to accurately reflect gene edits, according to new research led by scientists at Oregon Health & Science University.

7 Mar 2023

Fighting pancreatic cancer with gene and cell biotherapies

Pancreatic cancer is an incurable form of cancer, and gene therapies are currently in clinical testing to treat this deadly disease. A comprehensive review of the gene and cell biotherapies in development to combat pancreatic cancer is published in the peer-reviewed journal Human Gene Therapy.

6 Mar 2023

New delivery system based on lipid nanoparticles could increase the efficiency of gene therapy

Lipid nanoparticles have been used to encapsulate CRISPR-Cas9 and deliver it to cells in mice, where it was highly effective at knocking down expression of a target protein.

2 Mar 2023

Gene Therapy News and Research

Further Reading

Optimized genome-editing method opens the door to more effective treatment of genetic diseases

3D structure of a probable precursor to the CRISPR-Cas12 enzyme revealed

Using brain organoids to test gene therapies for a rare disease in children

Complex assembly process involved in DNA virus replication revealed

Research provides new insights into the underlying disease mechanisms for polycystic kidney disease

Utilizing a molecular syringe for the delivery of functional proteins into human cells with high efficiency

Riboswitch may be the key to advance gene therapy, regenerative medicine

Lab-made antibodies may be able to cure people infected with yellow fever

Mouse avatars could help design more effective and personalized therapies for multiple myeloma

FDA-approved drug keeps light-sensitive photoreceptors alive in lab models of LCA 10

Developers of gene therapies can utilize FDA guidance to prepare ODD or RPDD applications

Discovery suggests a new way to treat spinal muscular atrophy

Disruption of the FcRn–IgG interaction may make AAV-based gene therapies effective

Snapping at new biotech breakthroughs with Gator Pro

Study highlights a persistent life expectancy gap among patients living with sickle cell disease

Study provides strong support for AAV.NT-3 gene therapy in the management of sarcopenia

University of Utah and Tikkunlev partner to bring heart failure gene therapy innovation to the clinic

Common scientific method to analyze DNA in early human embryos fails to accurately reflect gene edits

Fighting pancreatic cancer with gene and cell biotherapies

New delivery system based on lipid nanoparticles could increase the efficiency of gene therapy

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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